545 © American Association of Pharmaceutical Scientists 2019 P. V

Total Page:16

File Type:pdf, Size:1020Kb

545 © American Association of Pharmaceutical Scientists 2019 P. V Index A Although, 524 Aberrant expression, 118 Alzheimer’s disease, 305, 326 Abluminal, 47 American type culture collection (ATCC), 537 Actin, 7 AMH, see Anti-Mullerian hormone (AMH) Actinic keratosis, 342 Aminolevulinic acid, 501 Actinomycetes, 485 Amino-triphenyl dicarboxylate-bridged Zr4+ Activated macrophages, 13 metal-organic framework nanoparticles Activation, 93 (NMOFs), 215 Activation functions (AFs), 89 Amphiregulin (AREG), 240 Active targeting, 467 β-Amyloid fibrils, 305 Adamantane–hyaluronic acid, 420 Anaplastic large cell lymphoma (ALCL), 219 Adamantane polyethylene glycol, 473 Anaplastic lymphoma kinase (ALK), 233 Adaptive immune responses, 328 Ancillary targets, 165 Adaptor proteins, 15 Androgen receptor (AR), 115 Adenocarcinoma, 231 Androgen receptor antagonists, 123 Adenomatous polyposis coli (APC), 188, 191 Androgen response elements (ARE), 116 Adenosine triphosphate (ATP), 243 Androgens, 120 Adherens junction, 179 Ang2 inhibitor (recombinant peptide-Fc-­ Adsorptive endocytosis, 49 fusion protein), 215 Advanced chemorefractory endometrial Angiogenesis, 53, 490, 529 cancers, 193 Angiogenesis factors, 189 Advanced epithelial ovarian, 131 Angiogenic paracrine factors, 490 Advanced gastric adenocarcinoma, 220 Anilinoquinazoline tyrosine kinase inhibitor, 242 Advanced glycation end products (AGE), 305 Annexin V, 493 Advanced/metastatic NSCLC, 258 Annexin V-FITC/propidium iodide assay, Adverse effects, 400 534–536 A glycoprotein hormone, 121 Antagonists, 287, 393 Agonists, 87 Antiangiogenic activity, 527 AIDS, 280 Antiangiogenic drugs, 215 Albumin, 63, 102 Antiapoptotic proteins, 188 Albumin nanoparticles, 254 Antibiotics, 341 ALK inhibitors, 257 Antibodies, 9, 59, 411, 464, 471 Alkaline, 356 Antibody-dependent cell-mediated Allosteric sites, 192 cytotoxicity (ADCC), 84 © American Association of Pharmaceutical Scientists 2019 545 P. V. Devarajan et al. (eds.), Targeted Intracellular Drug Delivery by Receptor Mediated Endocytosis, AAPS Advances in the Pharmaceutical Sciences Series 39, https://doi.org/10.1007/978-3-030-29168-6 546 Index Antibody-directed enzyme prodrug therapy ATPase inhibitors, 506 (ADEPT), 59 ATP-binding cassettes (ABC), 98 Antibody–drug conjugates (ADC), 59, 133 ATP-competitive, 251 Antibody-mediated cellular cytoxicity ATX, see Autotaxin (ATX) (ADCC), 242 Autoimmune, 414 Antibody-nanoshell conjugates, 192 diseases, 328 Anticancer drugs, 485 events, 257 Anticancer vaccines, 445 Autophosphorylation, 153, 208 Anti-CD antibodies, 393 Autotaxin (ATX), 175 Antidiabetic agents, 160 Axin-1/axin-2 (tumor suppressor proteins), 188 Antiestrogens, 124 Antifolates, 411 Antigen–adjuvant complexes, 339 B Antigen-presenting cells (APCs), 339 Bacillus Calmette-Guerin (BCG), 342 Antihuman TfR, 471 Bacterial flagella, 336 Anti-infective drugs, 340, 485 Bacterial flagellin, 330 Anti-mucin 1 aptamer (Apt), 206 Bacterial load, 509 Anti-Mullerian hormone (AMH), 202 Bactericidal, 540 Antineuroblastoma, 215 Bacteriostatic, 540 Antiprogestins, 90 Basal lamina, 47 Antiproliferative activity, 96 B-cell receptor (BCR), 390 Antiretroviral, 510 BCG, see Bacillus Calmette-Guerin Antisense oligonucleotides, 336 (BCG) Antitumor agents, 340 BC staging, 82 Anti-VEFG intraceptor, 254 Betacellulin (BTC), 240 Antiviral agents, 340 Biantennary oligosaccharides, 361 APC, see Adenomatous polyposis coli Binding affinity, 356 (APC) Bioavailability, 154 APCs, see Antigen-presenting cells (APCs) Biochemical, 502 Apolipoprotein, 51 Biodegradable, 468 Apolipoprotein A1, 159 Biological functions, 389 Apoptosis, 199, 492 Biomarkers, 313, 448 Apoptotic bodies, 493 Biotin, 371 Apoptotic cells, 300 Bladder cancer, 414, 501 Aprotinin, 53 Block polymers, 99 Apt, see Anti-mucin 1 aptamer (Apt) B lymphocytes, 325 Aqueous lecithin dispersions, 488 BMPs, see Bone morphogenetic proteins Arabinogalactan (AG), 362 (BMPs) Arachidonic acid, 246 BMP type II receptor, 210 Aromatase inhibitors, 88 200-μl Boiling double-distilled, 528 β-Arrestins, 174, 179 Bombesin receptors (BBR1, BBR2 and Arthritis, 342 BBR3), 236 ASGP-R, see Asialoglycoprotein receptors Bone morphogenetic proteins (ASGP-Rs) (BMPs), 202 Asialoglycoprotein receptors (ASGP-Rs), 204, Bone sialoprotein, 55 355, 372 Boron nitride nanoparticles, 418 Aspartic acid, 250 Bradykinin receptors, 237 Aspartic acid-arginine-tyrosine, 145 BRCA1 mutations, 91 Astrocytes, 47 BrdU, see Bromodeoxyuridine (BrdU) Atazanavir, 422 BrdU-labeled assay, 526–527 ATCC, see American type culture collection Bromodeoxyuridine (BrdU), 487 (ATCC) α-Bromophosphonate, 187 Atherosclerosis, 281 Bronchioloalveolar carcinoma, 233 Athymic nude BALB/c mice, 254 Burkholderia cenocepacia, 504 Index 547 C Cellular proliferation, 409, 487 Ca2+-dependent phospholipid-binding Cellular signals, 173 protein, 534 Cellular trafficking, 438 Calcium ion mobilization, 178 Cellular uptake, 504, 541 Ca2+ levels, 360 Central nervous system, 337 CAM, see Chorioallantoic membrane (CAM) Cerebral malaria, 280 Camptothecin, 371, 418 Cervical and ovarian cancers, 111 Cancer, 5 CFP-YFP, see Cyan fluorescent protein– metastasis, 490 yellow fluorescent protein (CFP-YFP) progression, 174 CFU, see Colony-forming unit (CFU) stage, 459 Chemo-endocrine therapy, 94 Cannabinoid receptors, 192 Chemokine receptors, 175 Canonical Wnt β-catenin pathway, 188 Chemokines, 273 Capillary blood vessels, 527 Chemorefractory ewing sarcoma, 219 Carbocyclic phosphothionate, 187 Chemotactic, 490 Carbohydrate recognition domain (CRD), 358 Chemotherapeutic agent, 503 Carbohydrates, 300 Chemotherapy, 58, 199, 259 Carbon nanoparticles, 418 Chimeric (mouse/human) monoclonal Carbon nanotubes, 312, 418 antibody, 244 Carcinogens, 233 Chitosan nanoparticles, 248, 417 Caspase-3, 161 Chlamydiae, 485 Caspase activation, 536, 537 Chloroquine, 12 Castration-resistant prostate cancer (CRPC), 114 Cholesterol, 51 Cathelicidins, 12 Cholesterol ester, 300 Cathepsin B, 180 Chondroitin sulfate, 391 Cationic proteins, 49 Chorioallantoic membrane (CAM), 490, 529 Cation-supplemented, 537 Chromatin-remodeling proteins, 188 Caveolae, 241 Chronic inflammatory bowel disease, 161 Caveolin, 7 Chronic lymphocytic leukemia (CLL), 395, Caveolin-1, 16 399, 414 Caveosomes, 16 Chronic myelogenous leukemia, 154 CC receptors, 273 Circular dorsal ruffles (CDR), 7 CD3 complex, 386, 387 Cisplatin, 415 CD4, 387 C-Kit, 144, 151 CD8, 387, 393 Clathrin, 7 CD8-T cells, 339 Clathrin-coated pits, 15 CD19, 388, 390 Clathrin-mediated endocytosis, 241 CD20, 388, 391 Cleft like structure, 460 CD36, 303 CLIC/GEEC, 19 CD44, 385, 389 Clinical and Laboratory Standards Institute CD45, 435 (CLSI), 537 CD68, 305 Clinical pilot trial, 163 CDKs, see Cell cycle-regulated kinases Clinical trials, 68, 93, 128 (CDKs) C-lobe, 460 Cell cycle analysis, 492, 532–533 Clostridium difficile toxin B (TcdB), 190 Cell cycle-regulated kinases (CDKs), 214 CLSI, see Clinical and Laboratory Standards Cell cycle regulation, 164 Institute (CLSI) Cell differentiation, 162 Cluster differentiation 44 (CD44), 236 Cell internalization, 6 Cluster of differentiation (CD) molecules, 330 Cell line-derived xenograft model, 495 CmAbs, 400 Cell-penetrating peptides (CPP), 22 C-MET receptor, 247 Cell programming, 164 C-Met tyrosine kinase receptor, 200 Cell proliferation, 276, 489, 526–527 CNS diseases, 473 Cell surface receptors, 173 Coexpression, 240 548 Index Cofilin, 10 Cylindramatosis protein (CYLD), 335 Coiled-coil, 300 Cysteine-rich domain (CRD), 187 Collagenous domain, 301 Cytokine receptor, 151 Collagens, 55 Cytoplasmic domain, 358 Collectins, 9 Cytoplasmic tail, 52 Colocalization, 507 Cytoskeletal function, 250 Colon cancer, 463 Cytosol, 464 Colonic endoscopic biopsies, 192 Cytosolic, 21, 506 Colony-forming unit (CFU), 509 Cytostatic, 199 Colony-stimulating factor 1 (CSF-1), 214 Cytotoxic drug, 95 Colorectal, 463 Cytotoxic T lymphocytes (CTLs), 387 Colorectal cancer, 143, 414 Cytotoxicity, 421 Combination therapy, 129, 254 Competitive binding, 473 Competitive inhibition, 251 D Competitive inhibition assay, 505 DA, see Dodecylamine (DA) Complement-mediated toxicity, 242 DAG, see Diacylglycerol (DAG) Complement receptors (CR), 10 Damage-associate molecular patterns Computed tomography (CT), 248 (DAMPs), 326 Computer-aided screenings, 335 DAMPs, see Damage-associate molecular Concancamycin-A, 12 patterns (DAMPs) Conditional transgenic models, 502 Daunorubicin, 415 Confirmed response rate (CRR), 256 Decatenation, 532 Control of substances hazardous to health Deep epidermis, 460 (COSHH), 533 Defensins, 12 Coordinate bond, 360 Delivery, 248 Copper nanoclusters, 421, 472 Dendrimers, 65, 99, 471 Co-receptors, 330, 390 Dendritic cells, 325, 421 Coronins, 10 Development, 459 COSHH, see Control of substances hazardous Dextran, 288, 362 to health (COSHH) Diacylglycerol (DAG), 178, 183 CpG motifs, 328 Diacyl lipopeptide, 327 CRD, see Carbohydrate recognition domain Diagnostics, 67 (CRD); Cysteine-rich domain (CRD) Dihydrofolate reductase enzyme, 411 Crithidia fasciculata, 532 5α-Dihydrotestosterone (5 α-DHT), 115 Crohn’s disease, 337 Dimerization, 120 Cryo-anesthetize, 529 Discoid, 101 CSF-1, see Colony-stimulating factor 1 Dishevelled (DSH), 191 (CSF-1) Disintegrin, 240 C-terminal, 436 Disulfide-bonded dimers, 208 CTLDs, see C-type lectin domains (CTLDs) Disulfide bonds, 436 C-type lectin domains (CTLDs), 434 Disulfide bridges, 52 C-type lectin superfamily, 434 Dlg1, see Drosophila disc large tumor Curcumin, 421, 498 suppressor (Dlg1) CXCL12, see C-X-C motif chemokine 12 DNA, 409 (CXCL12) aptamers, 209 C-X-C motif chemokine 12 (CXCL12), 143 hydrolysis, 527 CXC receptors,
Recommended publications
  • List Item Withdrawal Assessment Report for Folcepri
    20 March 2014 EMA/CHMP/219148/2014 Committee for Medicinal Products for Human Use (CHMP) Assessment report Folcepri International non-proprietary name: etarfolatide Procedure No.: EMEA/H/C/002570/0000 Note Assessment report as adopted by the CHMP with all information of a commercially confidential nature deleted. This AR reflects the CHMP opinion on 20 March 2014, which originally recommended to approve this medicine. The recommendation was conditional to the results of the on-going confirmatory study EC-FV-06. Before the marketing authorisation was granted by the EC, the results of this study became available and did not support the initial recommendation. Subsequently, the company decided to withdraw the application and not to pursue any longer the authorisation for marketing this product. The current report does not include the latest results of this study as the withdrawal of the application did not allow for the CHMP to revise its opinion in light of the new data. For further information please refer to the Q&A which followed the company’s withdrawal of the application. 30 Churchill Place ● Canary Wharf ● London E14 5EU ● United Kingdom Telephone +44 (0)20 3660 6000 Facsimile +44 (0)20 3660 5505 Send a question via our website www.ema.europa.eu/contact An agency of the European Union © European Medicines Agency, 2014. Reproduction is authorised provided the source is acknowledged. Table of contents 1. Background information on the procedure .............................................. 6 1.1. Submission of the dossier ...................................................................................... 6 1.2. Manufacturers ...................................................................................................... 8 1.3. Steps taken for the assessment of the product ......................................................... 8 2. Scientific discussion ...............................................................................
    [Show full text]
  • ESMO 2014 Scientific Meeting Report
    ESMO 2014 Congress Scientific Meeting Report – Lung Cancer Extract 26-30 September 2014 Madrid, Spain Summary The European Society for Medical Oncology (ESMO) Congress, held September 26 to 30 in Madrid, Spain, was a record-breaker on nearly all levels. It was resounding success and in a dedicated infographic you can find the congress statistics. A primary emphasis in the scientific programme was placed on precision medicine and how it will change the future treatment landscape in oncology. In addition, a number of scientific presentations were dedicated to cancer immunology and immunotherapy across multiple tumour types. This report is an overview of key scientific presentations made during the congress by leading international investigators. It attempts to represent the diversity and depth of the ESMO 2014 scientific programme, as well as advances in oncology. Infographic (right): ESMO 2014 record breaking Congress ESMO 2014 Congress Meeting Report Page 1 © Copyright 2014 European Society for Medical Oncology. All rights reserved worldwide. Contents Lung Cancer .................................................................................................................................... 3 Final results of the SAKK 16/00 trial: A randomised phase III trial comparing neoadjuvant chemoradiation to chemotherapy alone in stage IIIA/N2 NSCLC ................................................. 3 Adjuvant treatment with MAGE-A3 cancer immunotherapeutic in patients with resected NSCLC does not increase DFS: Results of the MAGRIT, a double-blind,
    [Show full text]
  • Esmo – Missed Target Leaves Endocyte Clutching at Straws
    September 28, 2014 Esmo – Missed Target leaves Endocyte clutching at straws Jacob Plieth Followers of Endocyte had pinned their hopes on overall survival data from vintafolide’s phase II Target study providing definitive proof of efficacy. With results presented at the Esmo congress on Saturday, however, defeat has been snatched from the jaws of victory. Even a broad data dredge has barely managed to tease out a subgroup benefit – after one accepts Target’s advantageous statistical design, that is. Endocyte had risen 6% on Thursday in anticipation of the late-breaking Esmo data, but when the markets open again tomorrow investors will almost certainly be left nursing losses. There were already question marks around vintafolide, Endocyte’s lead project. European approval was revoked after the Proceed study in ovarian cancer was halted for futility, prompting Merck & Co, which had paid $120m up front to access vintafolide, to pull out of the deal (Trial miss leaves Endocyte at the mercy of Merck, May 6, 2014). Back in March, progression-free survival (PFS) results from the Target trial, which pitched vintafolide and docetaxel against docetaxel alone in a second-line setting for NSCLC, caused Endocyte to surge, and the company managed to raise $102m from investors. The fate of vintafolide, which comprises vinblastine linked to a compound that targets the folate receptor, thus rested on further analyses of Target – most crucially of patients’ overall survival (OS). In fact, the OS data showed vintafolide scoring a miss. Risk of death was reduced by 12% – though with the upper confidence interval bound at 1.36 – with a p value of 0.2874.
    [Show full text]
  • (Ec145) and Pegylated Liposomal Doxorubicin (Pld/Doxil®/Caelyx®) in Combination Versus Pld in Patients with Platinum-Resistant Ovarian Cancer
    EC-FV-06 Clinical Study Report Page 1 1. EC-FV-06 Clinical Study Report A RANDOMIZED DOUBLE-BLIND PHASE 3 TRIAL COMPARING VINTAFOLIDE (EC145) AND PEGYLATED LIPOSOMAL DOXORUBICIN (PLD/DOXIL®/CAELYX®) IN COMBINATION VERSUS PLD IN PATIENTS WITH PLATINUM-RESISTANT OVARIAN CANCER Vintafolide (EC145): Targeted Therapeutic Agent 99mTc-etarfolatide: Companion Diagnostic Imaging Agent Therapy for platinum-resistant epithelial ovarian, fallopian tube or primary peritoneal cancer This was an international, multicenter, centrally-randomized, double-blind, Phase 3, two-arm study comparing EC145 + PLD and placebo + PLD, given until disease progression or unacceptable toxicity in patients platinum-resistant ovarian, fallopian tube, or primary peritoneal cancer Endocyte, Inc. Protocol EC-FV-06 (PROCEED) Phase 3 First patient enrolled (assigned to therapy): 22 April 2011 Date of early study termination: 20 May 2014 Data cutoff date: 17 March 2014 Approval Date: 15 February 2017 Responsible Medical Officer: Alison Armour MB.ChB., BSc., MSc., MD., MRCP., FRCR Endocyte, Inc. 8910 Purdue Road, Suite 250 Indianapolis, IN 46268 This study was performed in compliance with the principles of good clinical practice (GCP) and Endocyte, Inc. standard operating procedures. The information contained in this clinical study report is confidential and may not be reproduced or otherwise disseminated without the written approval of Endocyte, Inc.. This document and its associated appendices are subject to United States Freedom of Information Act (FOIA) Exemption 4. Vintafolide (EC145) EC-FV-06 Clinical Study Report Page 2 2. Synopsis Title of Study: A Randomized Double-Blind Phase 3 Trial Comparing Vintafolide (EC145) and Pegylated Liposomal Doxorubicin (PLD/DOXIL®/CAELYX®) in Combination Versus PLD in Patients with Platinum- Resistant Ovarian Cancer Number of Investigator(s): This multicenter study included 194 principal investigators.
    [Show full text]
  • Access to Cancer Medicines in Australia
    Access to cancer medicines in Australia Medicines Australia Oncology Industry Taskforce July 2013 Contents Glossary ..................................................................................................................................... i Executive summary .................................................................................................................... i 1 Background ..................................................................................................................... 1 1.1 Purpose of this report ....................................................................................................... 2 1.2 Methods ........................................................................................................................... 3 1.3 Report structure ............................................................................................................... 9 2 Cancer in Australia and other countries ......................................................................... 10 2.1 Population statistics on cancer ........................................................................................ 10 2.2 Population impacts of cancer in Australia ........................................................................ 24 2.3 Summary ........................................................................................................................ 33 3 Current and future cancer medicines ............................................................................ 34 3.1 Current
    [Show full text]
  • Annexes to the Annual Report of the European Medicines Agency 2014
    Annexes to the annual report of the European Medicines Agency 2014 Table of contents Annex 1 – Members of the Management Board ............................................................................. 2 Annex 2 – Members of the Committee for Medicinal Products for Human Use ................................... 4 Annex 3 – Members of the Pharmacovigilance Risk Assessment Committee ...................................... 6 Annex 4 – Members of the Committee for Medicinal Products for Veterinary Use ............................... 8 Annex 5 – Members of the Committee on Orphan Medicinal Products ............................................ 10 Annex 6 – Members of the Committee on Herbal Medicinal Products .............................................. 12 Annex 07 – Committee for Advanced Therapies .......................................................................... 14 Annex 8 – Members of the Paediatric Committee ........................................................................ 16 Annex 9 – Working parties and working groups .......................................................................... 18 Annex 10 – CHMP opinions in 2014 on medicinal products for human use ...................................... 22 Annex 11 – CVMP opinions in 2014 on medicinal products for veterinary use .................................. 36 Annex 12 – COMP opinions in 2014 on designation of orphan medicinal products ............................ 41 Annex 13 – HMPC European Union herbal monographs in 2014....................................................
    [Show full text]
  • Rational Combination Therapy of Vintafolide (EC145) with Commonly Used Chemotherapeutic Drugs
    Author Manuscript Published OnlineFirst on January 15, 2014; DOI: 10.1158/1078-0432.CCR-13-2423 Author manuscripts have been peer reviewed and accepted for publication but have not yet been edited. 1 Rational combination therapy of vintafolide (EC145) with commonly used chemotherapeutic drugs Joseph A. Reddy1, Ryan Dorton1, Alicia Bloomfield1, Melissa Nelson1, Marilynn Vetzel1, John Guan1 and Christopher P. Leamon1 Endocyte, Inc., 3000 Kent Ave, Suite A1-100, West Lafayette, IN 47906, USA 1Corresponding author: Dr. Christopher P. Leamon 3000 Kent Ave. Suite A1-100 West Lafayette, IN 47906 Phone: (765)463-7175 FAX: (765)463-9271 Email: [email protected] Running Title: Vintafolide drug combination studies Keywords: Folate receptor, targeted chemotherapy, ovarian cancer, EC145, vintafolide Downloaded from clincancerres.aacrjournals.org on September 30, 2021. © 2014 American Association for Cancer Research. Author Manuscript Published OnlineFirst on January 15, 2014; DOI: 10.1158/1078-0432.CCR-13-2423 Author manuscripts have been peer reviewed and accepted for publication but have not yet been edited. 2 TRANSLATIONAL RELEVANCE Our report highlights the potential for safely combining vintafolide (a folate-targeted vinca alkaloid) with pegylated liposomal doxorubicin (PLD), platinum-based agents, topoisomerase inhibitors and taxanes to yield enhanced antitumor effects, including complete responses and cures against human tumor xenografts. The observed therapeutic benefits of these combinations were specific for folate receptor (FR)-positive tumor models, likely because vintafolide remains associated with tumor cells for multiple M phases of the cell due to its natural high affinity binding to the FR and the slow recycling rate of that receptor. Based on the disclosed preclinical data, a randomized phase 2 trial (PRECEDENT) was conducted with the vintafolide/PLD combination.
    [Show full text]
  • Patent Application Publication ( 10 ) Pub . No . : US 2019 / 0192440 A1
    US 20190192440A1 (19 ) United States (12 ) Patent Application Publication ( 10) Pub . No. : US 2019 /0192440 A1 LI (43 ) Pub . Date : Jun . 27 , 2019 ( 54 ) ORAL DRUG DOSAGE FORM COMPRISING Publication Classification DRUG IN THE FORM OF NANOPARTICLES (51 ) Int . CI. A61K 9 / 20 (2006 .01 ) ( 71 ) Applicant: Triastek , Inc. , Nanjing ( CN ) A61K 9 /00 ( 2006 . 01) A61K 31/ 192 ( 2006 .01 ) (72 ) Inventor : Xiaoling LI , Dublin , CA (US ) A61K 9 / 24 ( 2006 .01 ) ( 52 ) U . S . CI. ( 21 ) Appl. No. : 16 /289 ,499 CPC . .. .. A61K 9 /2031 (2013 . 01 ) ; A61K 9 /0065 ( 22 ) Filed : Feb . 28 , 2019 (2013 .01 ) ; A61K 9 / 209 ( 2013 .01 ) ; A61K 9 /2027 ( 2013 .01 ) ; A61K 31/ 192 ( 2013. 01 ) ; Related U . S . Application Data A61K 9 /2072 ( 2013 .01 ) (63 ) Continuation of application No. 16 /028 ,305 , filed on Jul. 5 , 2018 , now Pat . No . 10 , 258 ,575 , which is a (57 ) ABSTRACT continuation of application No . 15 / 173 ,596 , filed on The present disclosure provides a stable solid pharmaceuti Jun . 3 , 2016 . cal dosage form for oral administration . The dosage form (60 ) Provisional application No . 62 /313 ,092 , filed on Mar. includes a substrate that forms at least one compartment and 24 , 2016 , provisional application No . 62 / 296 , 087 , a drug content loaded into the compartment. The dosage filed on Feb . 17 , 2016 , provisional application No . form is so designed that the active pharmaceutical ingredient 62 / 170, 645 , filed on Jun . 3 , 2015 . of the drug content is released in a controlled manner. Patent Application Publication Jun . 27 , 2019 Sheet 1 of 20 US 2019 /0192440 A1 FIG .
    [Show full text]
  • Folate Receptor: a Potential Target in Ovarian Cancer
    Pteridines 2015; 26(1): 1–12 Review Marie Bartouskova, Bohuslav Melichar and Beatrice Mohelnikova-Duchonova* Folate receptor: a potential target in ovarian cancer Abstract: Ovarian cancer is the most frequent cause of Introduction gynecological cancer-related death. Unfortunately, many patients are diagnosed at an advanced stage and have a Folic acid antagonists have been used in the treatment poor prognosis. The standard treatment for advanced dis- of cancer for more than six decades, since 1948 [1]. Folic ease involves maximal cytoreductive surgery and chemo- acid is a vitamin required for cell metabolism, DNA syn- therapy based on platinum compounds and taxanes. thesis, and repair [2]. Two cellular folate uptake pathways Patients presenting at an advanced stage have a higher have been characterized. The first is mediated by trans- risk of recurrence. The development of drug resistance membrane-reduced folate carriers, which represent the currently represents a major obstacle in the systematic predominant pathway for folate uptake by normal human treatment and, therefore, the discovery of new anticancer cells. Whereas in epithelial cancer cells, membrane pro- agents and approaches should improve the poor progno- teins called folate receptors are often highly expressed and sis of these patients. Folate receptor α is overexpressed in mediate the folate uptake by tumors. Epithelial ovarian epithelial ovarian cancer (EOC), but has limited expres- carcinoma (EOC) has the highest expression of these pro- sion in nonmalignant human tissues. The degree of folate teins, making the folate receptor an attractive candidate receptor expression corresponds with the stage and grade for targeted therapy and targeted drug delivery therapy in of the disease.
    [Show full text]
  • Orphan Drug Designation List
    Orphan Drug Designations and Approvals List as of 09‐01‐2015 Governs October 1, 2015 ‐ December 31, 2015 Row Contact Generic Name Trade Name Designation Date Designation Num Company/Sponsor 1 (‐)‐(3aR,4S,7aR)‐4‐Hydroxy‐4‐ m‐tolylethynyl‐octahydro‐ Novartis indole‐1‐carboxylic acid Pharmaceuticals methyl ester n/a 10/12/2011 Treatment of Fragile X syndrome Corp. 2 (1‐methyl‐2‐nitro‐1H‐ imidazole‐5‐yl)methyl N,N'‐ bis(2‐broethyl) diamidophosphate n/a 6/5/2013 Treatment of pancreatic cancer EMD Serono 3 (1‐methyl‐2‐nitro‐1H‐ imidazole‐5‐yl)methyl N,N'‐ bis(2‐bromoethyl) Threshold diamidophosphate n/a 3/9/2012 Treatment of soft tissue sarcoma Pharmaceuticals, Inc. 4 (1OR)‐7‐amino‐12‐fluoro‐ 2,10,16‐trimethyl‐15 oxo‐ 10,15,16,17‐tetrahydro‐2H‐8,4‐ Treatment of anaplastic (metheno)pyrazolo[4,3‐ lymphoma kinase (ALK)‐positive h][2,5,11]benzoxadiazacyclote or ROS1‐positive non‐small cell tradecine‐3‐carbonitrile n/a 6/23/2015 lung cancer Pfizer, Inc. 5 (1R,3R,4R,5S)‐3‐O‐[2‐O‐ Treatment of vaso‐occlusive benzoyl‐3‐O‐(sodium(2S)‐3‐ crisis in patients with sickle cell cyclohexyl‐propanoate‐ n/a 2/17/2009 disease. Pfizer, Inc. 6 (1S)‐1‐(9‐deazahypoxanthin‐9‐ yl)‐1,4‐dideoxy‐1,4‐imino‐D‐ Treatment of acute Mundipharma ribitol‐hydrochloride n/a 8/13/2004 lymphoblastic leukemia Research Limited Page 1 of 359 Orphan Drug Designations and Approvals List as of 09‐01‐2015 Governs October 1, 2015 ‐ December 31, 2015 Row Contact Generic Name Trade Name Designation Date Designation Num Company/Sponsor 7 Treatment of chronic lymphocytic leukemia and related leukemias to include (1S)‐1‐(9‐deazahypoxanthin‐9‐ prolymphocytic leukemia, adult T‐ yl)‐1,4‐dideoxy‐1,4‐imino‐D‐ cell leukemia, and hairy cell Mundipharma ribitol‐hydrochloride n/a 8/10/2004 leukemia Research Ltd.
    [Show full text]
  • WO2020264398A1.Pdf
    ( (51) International Patent Classification: (71) Applicant: NURIX THERAPEUTICS, INC. [US/US]; A61P35/00 (2006.01) C07D 413/14 (2006.01) 1700 Owens Street, Suite 205, San Francisco, California C07D 401/14 (2006.01) C07D 471/04 (2006.01) 94158 (US). C07D 403/10 (2006.01) A61P 37/00 (2006.01) (72) Inventors: SANDS, Arthur T.; c/o Nurix Therapeutics, C07D 403/12 (2006.01) A61K 31/506 (2006.01) Inc., 1700 Owens Street, Suite 205, San Francisco, Califor¬ C07D 405/14 (2006.01) nia 94158 (US). BENCE, Neil F.; c/o Nurix Therapeutics, (21) International Application Number: Inc., 1700 Owens Street, Suite 205, San Francisco, Califor¬ PCT/US2020/039957 nia 94158 (US). ZAPF, Christoph W.; c/o Nurix Thera¬ peutics, Inc., 1700 Owens Drive, Suite 205, San Francis¬ (22) International Filing Date: co, California 94158 (US). COHEN, Frederick; c/o Nurix 26 June 2020 (26.06.2020) Therapeutics, Inc., 1700 Owens Drive, Suite 205, San Fran¬ (25) Filing Language: English cisco, California 94158 (US). WANG, Chenbo; c/o Nurix Therapeutics, Inc., 1700 Owens Drive, Suite 205, San Fran¬ (26) Publication Language: English cisco, California 94158 (US). CUMMINS, Thomas; c/o (30) Priority Data: Nurix Therapeutics, Inc., 1700 Owens Drive, Suite 205, San 62/866,914 26 June 2019 (26.06.2019) US Francisco, California 94158 (US). TANAKA, Hiroko; c/ 62/880,285 30 July 2019 (30.07.2019) US o Nurix Therapeutics, Inc., 1700 Owens Drive, Suite 205, 62/888,845 19 August 2019 (19.08.2019) US San Francisco, California 94158 (US). SHUNATONA, 62/888,870 19 August 2019 (19.08.2019) US Hunter; c/o Nurix Therapeutics, Inc., 1700 Owens Street, (54) Title: SUBSTITUTED BENZYL-TRIAZOLE COMPOUNDS FOR CBL-B INHIBITION, AND FURTHER USES THEREOF FIG.
    [Show full text]
  • Updates in Cancer for Clinicians
    UPDATES IN CANCER FOR CLINICIANS WINTER 2014 Progress in Hematologic Cell Transplantation has been associated with lower myeloid leukemia in first remission.1 rates of relapse in acute myeloid With a median follow up of leukemia and better overall surviving patients exceeding five outcomes. These studies utilized years, the non-relapse mortality fixed oral doses of busulfan, of patients receiving IV busulfan which is associated with up to (IV Bu) was only 12 percent at twentyfold variations in plasma one year and 18 percent at five Edward Copelan, MD, (left) levels. Low plasma levels are years, comparable to reports using Chair of the Department of Hematologic associated with graft rejection and allegedly safer reduced intensity Oncology and Blood Disorders leukemia relapse and high levels regimens, and significantly better with toxicity and transplant-related than the TBI group in the study. Belinda Avalos, MD (right) Vice Chair of the Department of mortality. Improved methods Survival and leukemia free survival Hematologic Oncology and Blood of administration, including the (57 percent at five years for IV Disorders intravenous route and dose Bu) were significantly higher and adjustment of busulfan based on late relapse significantly less Initial studies demonstrating plasma levels, represent important frequent with IV Bu compared a cure of acute leukemia with advances which result in much to TBI. A supportive prospective allogeneic transplantation less variation in plasma levels cohort study in patients with were conducted by Nobel and less toxicity. Drs. Copelan various diagnoses, including Laureate E. Donnall Thomas. and Avalos pioneered the use of AML, and shorter follow up and Total body irradiation (TBI) with busulfan preparative regimens an accompanying commentary cyclophosphamide as preparative and advances in its administration.
    [Show full text]